The Federal Center for Brain and Neurotechnology of the Federal Medical and Biological Agency (FSBI FCBN of the FMBA) of Russia has the capacity to create its own therapy for the treatment of spinal muscular atrophy (SMA), Veronika Skvortsova, the head of the FMBA, said. According to her, the center plans to organize the necessary research, TASS reports.
“The technologies that are used in this center may enable us to create such a drug,” she said, answering the relevant question. “But, as you know, there is a long way to go, because it requires segregation and very complex clinical studies. It is true that such possibility exists. And I know that Vsevolod Vadimovich (Belousov, director of the center. – GxP News) plans to organize similar trials here.”
Spinal muscular atrophy is a rare genetic disease that occurs as a result of a mutation of survival of motor neuron 1 (SMN1), when the protein is produced in insufficient quantities or not at all. This protein supports the functions of motor neurons that control muscle movement. Its lack gradually leads to muscle atrophy, which can later cause disability or death. The expanded neonatal screening, which will be conducted in Russia starting from 2023, will also include this disease.
SMA occurs approximately in 1 per 7,000 newborns and is the most common cause of genetically determined infant mortality. The therapies for the disease are Zolgensma and Spinraza. The former is used once before the age of two years for type I SMA, the later is administered every four months.